The table below is a review of notable updates that occurred in June 2022 for investigational products in development (not an inclusive list). Click on the status link to view our full coverage.

Drug Pharmacologic Class Proposed Indication Status
Cardiovascular Disease
Landiolol (Eagle Pharmaceuticals, Inc.) Ultra-short acting, cardioselective, beta-1 adrenoreceptor blocker Short-term reduction of ventricular rate in patients with supraventricular tachycardia, including atrial fibrillation and atrial flutter. NDA submitted
Omecamtiv mecarbil (Cytokinetics, Inc.) Selective small molecule cardiac myosin activator Treatment of heart failure with reduced ejection fraction. PDUFA target date has been extended
Hematological Disorders
Betibeglogene autotemcel (beti-cel; bluebird bio, Inc.) One-time gene therapy that adds functional copies of a modified form of the β-globin gene into a patient’s own hematopoietic stem cells. Treatment of patients with beta-thalassemia who require regular red blood cell transfusion. FDA panel voted in favor of approval
Elivaldogene autotemcel (eli-cel; bluebird bio, Inc.) One-time gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells. Treatment of early active cerebral adrenoleukodystrophy in patients less than 18 years of age who do not have an available and willing HLA-matched sibling HSC donor. FDA panel voted in favor of approval
VLX-1005 (Veralox Therapeutics) Selective small molecule 12-LOX inhibitor Treatment and prevention of heparin-induced thrombocytopenia and thrombosis. Fast Track designation
Immunization
NVX-CoV2373 (Novavax) Protein-based COVID-19 vaccine Active immunization for the prevention of COVID-19 for individuals 18 years of age and older. FDA panel voted in favor of granting EUA
Infectious Diseases
Nirmatrelvir + ritonavir (Paxlovid; Pfizer Inc.) SARS-CoV-2 main protease inhibitor + HIV-1 protease inhibitor Treatment of COVID-19 in both vaccinated and unvaccinated individuals who are at high risk for progression to severe illness from COVID-19. NDA submitted
Tebipenem HBr (tebipenem pivoxil hydrobromide; Spero Therapeutics) Oral antibiotic in carbapenem class Treatment of adults with complicated urinary tract infections, including pyelonephritis, caused by susceptible microorganisms. Complete Response Letter issued
Musculoskeletal Disorders
BCX9250 (BioCryst Pharmaceuticals, Inc.) Activin receptor-like kinase-2 (ALK-2) inhibitor Prevention of heterotopic ossification in patients with fibrodysplasia ossificans progressiva. Fast Track designation
Neurologic Disorders
AMX0035 (sodium phenylbutyrate and taurursodiol; Amylyx Pharmaceuticals, Inc.) Oral, fixed-dose coformulation targeting endoplasmic and mitochondrial-dependent neuronal degeneration pathways Treatment of amyotrophic lateral sclerosis (ALS). PDUFA target date has been extended
ATX01 (topical amitriptyline; AlgoTx) Directly inhibits pain signaling in the nerve fibers on the epidermis and dermis Treatment of chemotherapy-induced neuropathic pain. Fast Track designation
Pimavanserin (Acadia Pharmaceuticals Inc.) Atypical antipsychotic Treatment of hallucinations and delusions associated with Alzheimer disease psychosis. FDA panel voted against approval
Ublituximab (TG Therapeutics, Inc.) Glycoengineered monoclonal antibody targeting CD20 Treatment of patients with relapsing forms of multiple sclerosis. PDUFA target date has been extended
Ob/Gyn
Myfembree (relugolix, estradiol, and norethindrone acetate; Myovant Sciences) GnRH antagonist + estrogen + progestin To include long term data in premenopausal women with heavy menstrual bleeding associated with uterine fibroids. sNDA accepted for review
Oncology
Omburtamab (Y-mAbs Therapeutics, Inc.) Radiolabeled monoclonal antibody that targets tumor cells expressing B7-H3 Treatment of pediatric patients with CNS/leptomeningeal metastasis form neuroblastoma BLA accepted for Priority Review
VBI-1901 (VBI Vaccines Inc.) Bivalent eVLP vaccine immunotherapeutic candidate that targets 2 highly immunogenic CMV antigens, gB and pp65. Treatment of glioblastoma. Orphan Drug designation
Ophthalmic Disorders
Aflibercept (Eylea Regeneron Pharmaceuticals, Inc.) Recombinant fusion protein (human VEGF inhibitor + human IgG1) Treatment of diabetic retinopathy with every 16-week dosing regimen. sBLA accepted for review
Respiratory Disorders
AD109 (atomoxetine + aroxybutynin; Apnimed) SNRI + selective antimuscarinic Treatment of obstructive sleep apnea. Fast Track designation